PTC Therapeutics is on track to file for approval of a new therapy for rare disease phenylketonuria after a positive phase 3 readout that could extend its product portfoli
The EMA's human medicines advisory committee has recommended that BioMarin Pharmaceutical's haemophilia A gene therapy Roctavian be approved in the EU, removing one of the last barriers to
In 2015, NICE said that BioMarin's Vimizim could be used to treat patients with the ultra-rare disease Morquio A syndrome, while real-world data was collected on its efficacy.
BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PK
US biotech BioMarin has hit back with a filing for a rare disease drug, after suffering what was described by analysts as a “major setback” when the FDA rejected its gene therapy for haemop
MSD and partner Moderna have started a late-stage trial of their personalised cancer vaccine V940 as a combination with MSD’s PD-1 inhibitor Keytruda for patients with non
On 1st October, the roll-out of the National Contract Value Review (NCVR) process marked a pivotal moment in the trajectory of clinical research in the UK, introducing a standardised, natio